Compassionate Use and Expanded Access
A patient with:
- advanced disease
- no approved treatment options
- no appropriate clinical trial options
may consider trying to get a new, unapproved drug outside of the clinical trial.
Access to a scientifically-tested drug outside of a clinical trial and prior to FDA approval has many names but is most commonly referred to as compassionate use.
When a drug company decides to provide access to their unapproved drug outside of the clinical trial they most commonly do it in the following two ways:
Expanded Access Programs (EAP)top of the page
Drug companies in the late stages of drug development including the Phase III clinical trial stage, can offer Expanded Access Programs for patients who are not able to enroll in a clinical trial. The FDA generally approves these programs if the drug has demonstrated some effectiveness against a specific cancer in the on-going clinical trials.
Single Patient Accesstop of the page
Patients who are not eligible for either clinical trials or an Expanded Access Program (if one exists) may be eligible to receive the unapproved new drug by applying for Single Patient Access. In this situation, the patient's doctor must first request permission for access to the drug from the drug company. If the company agrees, the patient's doctor works with the drug company to apply for FDA approval for use of the drug for the patient. The timeline for Single Patient Access varies. In an emergency, the FDA can walk the paperwork through in 24 hours.
Check Meeting FDA Requirements for Single Patient Access for more details on the Single Patient Access process.
Cautionary Notestop of the page
- Prepare to be confused: There are several regulatory mechanisms (programs) along with multiple terms and definitions that are used to describe how a patient may get access to an unapproved new drug outside of a clinical trial. We will define terms used on this web site but we want you to understand that drug companies, patient advocacy organizations and the FDA may use different terms.
For example, the FDA terminology is specified in the FDA regulations. Most drug companies refer to their compassionate access programs with terms that are unique to their specific program and inconsistent with the FDA language. In short, the definitions and terms can be confusing.
- The simplest way to get access to an unapproved drug is through a clinical trial. Expanded access programs, if offered by the drug company, offer another option. If neither of those two options exists, access through single-patient compassionate use may be possible.
- Working out single-patient compassionate use of an unapproved drug will be confusing, time-consuming and frustrating. There is no single policy or process adhered to by the FDA and drug companies. There is no single list of drugs currently available through compassionate use. There is no way to force the drug company or FDA to permit treatment through compassionate use.
Drug Company and FDA Approval Required for Compassionate Usetop of the page
Before a patient or group of patients is allowed to have access to an unapproved, new drug outside of a clinical trial two things MUST be in place:
- The owner (sometimes referred to as the sponsor, generally a drug company) of the new, unapproved drug must agree to allow the use of their drug outside of the clinical trial, and
- The FDA oncology medical officer in charge of the regulatory oversight of that new drug's development must concur before the patient or patients may begin taking the drug.
Many drug companies refuse compassionate use requests. Reasons for refusal may include:
- Lack of efficacy - there may be little evidence that the drug may be effective for the specific illness
- Safety - the drug may have toxicity problems which aren't public knowledge.
- Cost - compassionate use can be expensive, especially large access programs
- Production capacity - drugs can be expensive and time-consuming to make, and until approved, capacity to manufacture the drugs may be limited
- Priorities - companies may want to use 'left-over' drugs for clinical trials in different illnesses
It is important to remember that drug companies are not required to provide their unapproved drug to patients outside of a clinical trial and under no circumstances can a drug company be compelled to do so. The FDA generally supports requests for compassionate use if the patient has exhausted standard treatment options and is ineligible for clinical trials. However, if the FDA is aware of safety issues that may foreshorten a patient's life the FDA may refuse the patient's application. This is a rare occurrence.